How Clinical Trials Work in India?

Clinical trials are a key part of medical research. They help doctors and scientists find out whether a new medicine, treatment, or medical device is safe and effective for humans. In India, clinical trials follow specific methods to ensure the health and rights of participants are protected while producing accurate, reliable results. 

Before any new medicine can be made available to the public, it must go through a series of steps to validate safety and efficacy – the fundamentals of a clinical trial. These steps are carefully planned in what’s known as a ‘study design’. The goal is to prove that a treatment works better than placebo or existing treatments, and is safe to use across different populations.

The study design is the heart of a clinical trial. It outlines exactly how the research will be conducted – what is being tested, where are the tests being conducted, who will participate, how they will be divided into groups, and how the results will be measured. A well-thought-out design ensures that the data collected is meaningful and that the study is fair, unbiased, and safe for all participants.

• Randomised Controlled Trial (RCT): Participants are randomly assigned to groups - one gets the treatment, the other a placebo or existing ‘standard-of-care’ medicine. Ensures fairness and reliable results.

• Double-Blind Trial: Neither participants nor doctors know who’s getting the treatment or placebo. Removes bias.

• Open-Label Trial: Both doctor and participant know the treatment. Used when blinding isn’t feasible.

• Parallel-Arm Study: Two or more groups receive different treatments at the same time. Helps compare effectiveness directly.

• Crossover Study: Each participant receives multiple treatments in sequence. Useful when treatment effects are temporary.

• Multicentric Trial: Conducted at multiple hospitals or centres. Increases diversity and generalisability of results.

• Placebo-Controlled Study: One group gets the active treatment under study, the other gets an inactive placebo. Helps identify the real effects of the drug.

• Adaptive Trial Design: Allows modifications mid-study based on early data (e.g., expanding sample size). Increases efficiency and responsiveness.

• Single-Arm Study: All participants receive the same treatment. Often used in rare or life-threatening conditions where a control group isn't ethical.

• Interventional Study: Researchers actively assign treatment or interventions to participants across different groups.

• Observational Study: No treatment groups are assigned. Researchers observe and record treatment outcomes over time, without cross-group comparisons.

Each of these study designs is chosen based on the goals of the trial, the nature of the disease, the treatment being studied, and the need to ensure safety, fairness, and scientific accuracy.

In India, selecting participants for clinical trials is done very carefully. Each trial has inclusion and exclusion criteria, rules that determine who can or cannot join the study, based on age, health status, medical history, and other factors.

Before joining, every participant must go through an informed consent process. This means they are fully informed about the trial’s purpose, procedures, possible side effects, and their rights. Participation is completely voluntary, and people can choose to leave the trial at any point without affecting their medical care.

Clinical trials are conducted in four main phases, each with a specific purpose:

• Phase 1: Small group of healthy volunteers; checks basic safety and correct dosage.

• Phase 2: A Larger group of patients; tests how well the treatment works and looks for side effects.

• Phase 3: Large-scale testing on hundreds or thousands of patients; comparison with standard treatments.

• Phase 4: Post-approval monitoring; tracks long-term effectiveness and rare side effects in the general population.

  

Throughout the trial, doctors and researchers carefully monitor each participant. They collect medical data, track side effects, and measure the treatment’s effects through tests and observations.

Data is stored securely using digital systems. Independent review boards and monitoring committees check that the trial is progressing safely and ethically. If any serious issues arise, they can stop the trial immediately to protect participants.

India’s clinical trials follow a strong regulatory framework to ensure ethical conduct, transparency, and participant safety.

• The Central Drugs Standard Control Organisation (CDSCO) regulates trials under the Drugs and Cosmetics Act, 1940 and Rules, 1945.

• The New Drugs and Clinical Trials Rules (2019) introduced streamlined approvals, participant protections, and mandatory compensation for trial-related harm.

• Trials need clearance from both CDSCO and a registered Ethics Committee, ensuring scientific and ethical soundness.

• All trials must be registered with the Clinical Trials Registry – India (CTRI), maintained by ICMR, for public transparency.

India’s clinical research regulations have become stronger over time. The New Drugs and Clinical Trials Rules, 2019, introduced faster approval timelines, mandatory ethics approvals, and clear guidelines for compensating participants in case of injury or death during a trial.

These reforms have made India a more trusted and globally respected location for ethical clinical research. By understanding how clinical trials work, we can all play a role in supporting medical progress and improving healthcare for everyone.